Advertisement

Topics

Orphan drugs: trends and issues in drug development.

08:00 EDT 12th April 2018 | BioPortfolio

Summary of "Orphan drugs: trends and issues in drug development."

Research in rare diseases has contributed substantially toward the current understanding in the pathophysiology of the common diseases. However, medical needs of patients with rare diseases have always been neglected by the society and pharmaceutical industries based on their small numbers and unprofitability. The Orphan Drug Act (1983) was the first serious attempt to address the unmet medical needs for patients with rare diseases and to provide impetus for the pharmaceutical industry to promote orphan drug development. The process of drug development for rare diseases is no different from common diseases but involves significant cost and infrastructure. Further, certain aspect of drug research may not be feasible for the rare diseases. The drug-approving authority must exercise their scientific judgment and ensure due flexibility while evaluating data at various stages of orphan drug development. The emergence of patent cliff combined with the government incentives led the pharmaceutical industry to realize the good commercial prospects in developing an orphan drug despite the small market size. Indeed, many drugs that were given orphan designation ended up being blockbusters. The orphan drug market is projected to reach $178 billion by 2020, and the prospects of research and development in rare diseases appears to be quite promising and rewarding.

Affiliation

Journal Details

This article was published in the following journal.

Name: Journal of basic and clinical physiology and pharmacology
ISSN: 2191-0286
Pages:

Links

DeepDyve research library

PubMed Articles [21875 Associated PubMed Articles listed on BioPortfolio]

Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis.

In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we ...

Early Drug Development in the era of Immuno-Oncology: are we ready to face the challenges?

The classical development of drugs has progressively faded away, and we are currently in an era of seamless drug-development, where first-inhuman trials include unusually big expansion cohorts in the ...

Is the pharmaceutical industry's preoccupation with the monotherapy drug model stifling the development of effective new drug therapies?

Drug discovery and development is heavily biased towards the development of monotherapies. Screening, testing, and evaluation of mono-entity drugs are generally much simpler than drug combinations, an...

The drug repurposing landscape from 2012 to 2017: evolution, challenges, and possible solutions.

As the name suggests, drug repurposing is a strategy to identify new therapeutic uses for marketed drugs, discontinued and/or shelved drugs, and drug candidates currently in clinical development. Alth...

Progress in Inherited Retinal Disease Drug Discovery and Development: A Foundation's Perspective.

Ophthalmic drug discovery and development has enjoyed a recent renaissance, with a major shift away from reformulating old systemic drugs for ocular use to de novo discovery of drugs for specific ocul...

Clinical Trials [6676 Associated Clinical Trials listed on BioPortfolio]

FS-1 Drug for Treatment of Multiple Drug-resistant Tuberculosis

Drug-resistant tuberculosis is becoming a major problem for the whole humanity, and development of new Anti-TB drugs is of great importance. Since Kazakhstan is a country with the high bur...

Development of In Vitro Functional Assays From Primary Cells of Patients With Monogenic Diseases

The objective of this project is to build a collection of biological samples consisting of fibroblasts and primary keratinocytes from patients with orphan monogenic diseases and to use the...

Bioequivalence Study of Levothyroxine Sodium Tablets 150 mcg

The objective of this study is to establish the bioequivalence of two levothyroxine formulations through the estimation of T4 levels in serum samples after baseline correction, according t...

Sixteen Years Trends in Reported Undernutrition in Switzerland

To assess 16-year trends in undernutrition reporting and management in Switzerland

Tobacco Control-Related Policy Survey

Aim: to collect further information on issues related to tobacco control which should be addressed and advocated in further tobacco control measures in Hong Kong The objectives of this po...

Medical and Biotech [MESH] Definitions

Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

Drugs and their metabolites which are found in the edible tissues and milk of animals after their medication with specific drugs. This term can also apply to drugs found in adipose tissue of humans after drug treatment.

The use of multiple drugs administered to the same patient, most commonly seen in elderly patients. It includes also the administration of excessive medication. Since in the United States most drugs are dispensed as single-agent formulations, polypharmacy, though using many drugs administered to the same patient, must be differentiated from DRUG COMBINATIONS, single preparations containing two or more drugs as a fixed dose, and from DRUG THERAPY, COMBINATION, two or more drugs administered separately for a combined effect. (From Segen, Dictionary of Modern Medicine, 1992)

An orphan nuclear receptor that is implicated in regulation of steroidogenic pathways. It is unlike most orphan nuclear receptors in that it appears to lack an essential DNA-binding domain and instead acts as a transcriptional co-repressor. Mutations in the gene Dax-1 cause congenital adrenal hypoplasia.

Control of drug and narcotic use by international agreement, or by institutional systems for handling prescribed drugs. This includes regulations concerned with the manufacturing, dispensing, approval (DRUG APPROVAL), and marketing of drugs.

Advertisement
Quick Search
Advertisement
Advertisement

 


DeepDyve research library

Relevant Topics

Pharmacy
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...

Drug Discovery
Clinical Approvals Clinical Trials Drug Approvals Drug Delivery Drug Discovery Generics Drugs Prescription Drugs In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which drugs are dis...


Searches Linking to this Article